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Optimizing Hydroxyurea Treatment for Sickle Cell Disease Patients: The Pharmacokinetic Approach.

ABSTRACT: BACKGROUND:Hydroxyurea (HU) is a FDA- and EMA-approved drug that earned an important place in the treatment of patients with severe sickle cell anemia (SCA) by showing its efficacy in many studies. This medication is still underused due to fears of physicians and families and must be optimized. METHODS:We analyzed our population and identified HU pharmacokinetic (PK) parameters in order to adapt treatment in the future. Working with a pediatric population, we searched for the most indicative sampling time to reduce the number of samples needed. RESULTS:Nine children treated by HU for severe SCA were included for this PK study. HU quantification was made using a validated gas chromatography/mass spectrometry (GC/MS) method. Biological parameters (of effectiveness and compliance) and clinical data were collected. None of the nine children reached the therapeutic target defined by Dong et al. as an area under the curve (AUC) = 115 h.mg/L; four patients were suspected to be non-compliant. Only two patients had an HbF over 20%. The 2 h sample was predictive of the medication exposure (r2 = 0.887). CONCLUSIONS:It is urgent to be more efficient in the treatment of SCA, and pharmacokinetics can be an important asset in SCA patients.

SUBMITTER: Nazon C

PROVIDER: S-EPMC6833033 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature

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Optimizing Hydroxyurea Treatment for Sickle Cell Disease Patients: The Pharmacokinetic Approach.

Nazon Charlotte C Sabo Amelia-Naomi AN Becker Guillaume G Lessinger Jean-Marc JM Kemmel Véronique V Paillard Catherine C

Journal of clinical medicine 20191016 10

<h4>Background</h4>Hydroxyurea (HU) is a FDA- and EMA-approved drug that earned an important place in the treatment of patients with severe sickle cell anemia (SCA) by showing its efficacy in many studies. This medication is still underused due to fears of physicians and families and must be optimized.<h4>Methods</h4>We analyzed our population and identified HU pharmacokinetic (PK) parameters in order to adapt treatment in the future. Working with a pediatric population, we searched for the most ...[more]

PMID: 31623213

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Project description:Background and Objectives: Hydroxyurea is a crucial treatment for sickle cell disease (SCD), but some patients' adherence to it remains suboptimal. Understanding patients' perspectives on SCD and HU is essential for improving adherence. This study aimed to assess hydroxyurea adherence and patients' perceptions of SCD and hydroxyurea among SCD patients in the Jazan region of Saudi Arabia. Materials and Methods: This cross-sectional study collected data from 217 SCD patients using self-administered questionnaires from August 2022 to January 2023. The survey covered patient demographics, SCD consequences, and other clinical data. We used the Brief Illness Perception Questionnaire (B-IPQ) to measure patients' disease perception and the 8-item Morisky Medication Adherence Scale (MMAS-8) to evaluate patients' adherence to HU. Data were analysed using descriptive, t-test, and chi-square tests, and the p-value was set at <0.05 for significance. Results: More than half of the patients were male, with a mean age of 28.09 ± 8.40 years. About 57.6% of the patients were currently using HU. About 81.6% of HU users reported low adherence. The adherence was lower among individuals with infections/recurrent infections and in patients who received repeated blood transfusions. ICU admission, blood transfusion, and certain SCD complications were associated with HU use. Male patients had a higher perception of SCD consequences, concern, and understanding. ICU-admitted and recurrent hospitalized patients had a higher perception of the SCD-related consequences, symptoms, concerns, and emotional responses. Conclusions: HU seems a well-established and efficacious disease-modifying agent, but its underutilization for SCD patients remains challenging. To overcome the adherence challenges, healthcare providers must educate SCD patients about the role of hydroxyurea in lowering disease severity and addressing side effects to obtain maximum benefits. Healthcare providers may consider tailored educational interventions to improve adherence, particularly for patients with infections, recurrent hospitalizations, or repeated blood transfusions. Further research is needed to identify strategies for improving hydroxyurea adherence and patient education among SCD patients.

Dataset Information

Optimizing Hydroxyurea Treatment for Sickle Cell Disease Patients: The Pharmacokinetic Approach.

Publications

Optimizing Hydroxyurea Treatment for Sickle Cell Disease Patients: The Pharmacokinetic Approach.

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